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Pipeline Report
November 2021
• Recent COVID-19 updates
• FDA approvals for rare
disease therapies
• Upcoming gene therapy
developments
Pipeline Report This quarterly publication is developed by our Clinical Pharmacy Drug Information
team to increase your understanding of the drug pipeline, ensuring that you are
equipped with insights to prepare for shifts in prescription drug management.
February 2021
November 2021
© 2021 Envolve. All rights reserved. | Data is current as of 11/19/2021 Page 1Pipeline Report
Table of Contents During the past quarter, progress continued on
the COVID-19 vaccine front. All three currently
population beyond
that which is covered
November 2021
available vaccines have received Emergency by Kymriah, which is
Use Authorization (EUA) from the FDA for use approved for use in
as heterologous booster doses in adults after patients who are up
1 COVID-19 completion of a primary vaccination series. The to 25 years of age with
Pfizer vaccine was granted an EUA expansion for the same diagnosis.
use in children as young as five years of age, from
13 Recent Specialty Drug Approvals a previous authorization of at least 12 years of age. We continue to
In contrast, however, the FDA has decided to delay monitor developments
its decision on the use of the Moderna vaccine in of pipeline gene therapies, and 2022 promises
17 Recent Non-Specialty Drug Approvals adolescents 12 years of age and older in order to to be a boon for some of these. Signs point to
review its myocarditis risk in this population. The a BLA submission for AMT-061 (etranacogene
decision will likely be delayed until at least January dezaparvovec) for the treatment of hemophilia B
2022. Distribution of bamlanivimab + etesevimab in or around the first quarter of 2022. In addition, a
18 Upcoming Specialty Products resumed following data which showed that the BLA resubmission for Roctavian (valoctocogene
combination is active against the Delta variant, roxaparvovec) for hemophilia A is expected in the
and shortly thereafter it received an EUA for use as second quarter, with potential FDA approval of
32 Upcoming Non-Specialty Products post-exposure prophylaxis. An FDA authorization of both before the end of 2022. As for ciltacabtagene
molnupiravir would be a significant addition to the autoleucel for relapsed or refractory multiple
COVID therapeutics armamentarium, as it would be myeloma, an FDA decision date has been pushed
34 Biosimilars the first oral antiviral therapy aimed at treatment out until February 2022 to allow for review of
of adults with mild to moderate COVID-19 who are additional data submitted by the manufacturer.
at high risk for progression to severe disease. The An eventual FDA approval will add an anti-BCMA
37 Generic Specialty Agents EUA request has been submitted to the FDA with a CAR-T therapy option other than Abecma in this
decision expected as early as December. Even more therapeutic space.
cause for optimism is that Paxlovid is following
close behind, another oral antiviral therapy that Our pipeline report is just one of many ways we are
38 Generic Non-Specialty Agents appears highly effective at reducing severe COVID committed to providing helpful tools and resources
19-related disease and death. to our clients and partners. We look forward to
sharing more updates with you in the months
39 Glossary In non-COVID-19 news, one notable FDA approval ahead.
this past quarter is Rethymic (allogeneic
processed thymus tissue–agdc), the first
regenerative tissue-based therapy for immune
reconstitution in pediatric patients with congenital
athymia. This disease state had previously only Ross Hoffman, MD
been treatable with supportive care and typically
resulted in death by age two or three. Another To provide comments, feedback or requests
notable approval is a new indication for existing for report enhancements, please email
CAR T-cell therapy Tecartus (brexucabtagene us at Communications@EnvolveHealth.com.
autoleucel) in the treatment of adults with relapsed
or refractory acute lymphoblastic leukemia (ALL).
This new indication extends the treatable ALL
© 2021 Envolve. All rights reserved. | Data is current as of 11/19/2021 Page 2COVID-19
Pipeline Report
November 2021
FDA-APPROVED AGENTS
FDA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 10/22/2020 - FDA-approved for the treatment of adults and pediatric patients
≥ 12 years of age and weighing ≥ 40 kg requiring hospitalization for COVID-19 $520 for a 100 mg
EUA:
single dose vial
Veklury 5/1/2020 • 5/1/2020 - Veklury is available through an FDA Emergency Use Authorization
$3,120 for a 5-day
remdesivir Gilead FDA Treatment - antiviral (EUA) for the treatment of COVID-19 in hospitalized pediatric patients weighing
course
intravenous infusion Approval: 3.5 kg to < 40 kg or < 12 years of age and weighing ≥ 3.5 kg
$5,720 for a 10-day
10/22/2020 • Veklury should only be administered in a hospital or healthcare setting course
capable of providing acute care comparable to inpatient hospital care
*Initially, the federal government will supply the vaccine at no cost.
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November 2021
AGENTS GRANTED FDA EMERGENCY USE AUTHORIZATION (EUA)
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 11/19/2021: FDA amended the EUA to authorize use of a single booster dose for
all individuals 18 years of age and older after completion of primary vaccination
with any FDA-authorized or approved COVID-19 vaccine
• 10/29/21: FDA expanded the EUA to include use for prevention of COVID-19 in
individuals 5-11 years of age
• 10/20/2021: A single booster dose may be administered as a heterologous
Original EUA: booster dose following completion of primary vaccination with another
12/11/2020 authorized COVID-19 vaccine
Expanded • The eligible population(s) and dosing interval for the heterologous booster
EUA: dose are the same as those authorized for a booster dose of the vaccine
5/10/2021, used for primary vaccination
Comirnaty 8/12/2021, • 9/22/21: FDA expanded the EUA to include booster shots in individuals — 65
Pfizer COVID-19 vaccine Pfizer 9/22/2021, Vaccine years of age and older, 18–64 years of age at high risk of severe COVID-19, and $19.50 per dose*
intramuscular injection 10/20/2021, 18–64 years of age with frequent institutional or occupational exposure to
10/29/2021, SARS-CoV-2
11/19/2021 • 8/23/2021: FDA granted full FDA approval for the 2-dose regimen for active
immunization to prevent COVID-19 caused by SARS-CoV-2 in individuals 16
Full FDA years of age and older (use in people 12-15 years of age, and use of a third dose
approval: for immunocompromised people, are still authorized under the previous EUA)
8/23/2021
• 8/12/2021: FDA expanded the existing EUA to include a third dose in certain
immunocompromised people
• 5/10/2021: FDA expanded the existing EUA to include adolescents 12-15 years
of age, amending the EUA originally issued on 12/11/2020 for administration in
individuals 16 years of age and older
• No cost to patients
*Initially, the federal government will supply the vaccine at no cost.
CONTINUED
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November 2021
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 11/19/2021: FDA amended the EUA to authorize use of a single booster dose
for all individuals 18 years of age and older after completion of primary
vaccination with any FDA-authorized or approved COVID-19 vaccine
• 10/20/2021: EUA expansion for booster dose as follows:
• A single Moderna COVID-19 Vaccine booster dose (0.25 mL) may be
administered IM at least six months after completing a primary series
of the Moderna COVID-19 Vaccine to individuals — 65 years of age and
older, 18-64 years of age at high risk of severe COVID-19, and 18-64
years of age with frequent institutional or occupational exposure to
SARS-CoV-2
• A single booster dose of the Moderna COVID-19 Vaccine (0.25 mL) may
be administered as a heterologous booster dose following completion
12/18/2020 of primary vaccination with another authorized or approved COVID-19
vaccine
Moderna COVID-19 Expanded • The eligible population(s) and dosing interval for the heterologous booster
Vaccine EUA: dose are the same as those authorized for a booster dose of the vaccine
Moderna Vaccine $15-37 per dose*
mRNA 1273 8/12/2021, used for primary vaccination
intramuscular injection 8/30/2021,
• 10/15/2021: FDA is delaying its decision on the use of the Moderna
10/20/2021,
COVID-19 vaccine in adolesents 12-17 years of age, in order to assess the
11/19/2021
potential risk of myocarditis in this population
• 8/30/2021: Warning added re: postmarketing data demonstrating
increased risks of myocarditis and pericarditis (observed risk is higher
among males under 40 years of age), particularly within seven days
following the second dose
• 8/12/2021: FDA expanded the existing EUA to include a third dose in
certain immunocompromised people
• 6/1/2021: Moderna initiated rolling submission of BLA for full FDA licensure
in individuals 18 years of age and older
• 6/10/2021: Moderna filed for EUA expansion in adolescents ages 12-17;
mRNA vaccine that expresses SARS-CoV2 spike protein
• No cost to patients
*Initially, the federal government will supply the vaccine at no cost.
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November 2021
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 10/20/2021: Janssen vaccine received expanded EUA for use as a booster
dose:
• A single Janssen COVID-19 Vaccine booster dose (0.5 mL) may be
administered at least 2 months after primary vaccination with the
Janssen COVID-19 Vaccine, to individuals 18 years of age and older
• A single booster dose of the Janssen COVID-19 Vaccine (0.5 mL) may
be administered as a heterologous booster dose following completion
of primary vaccination with another authorized or approved COVID-19
vaccine; the eligible population(s) and dosing interval for the
Original:
heterologous booster dose are the same as those authorized for a
2/27/2021
booster dose of the vaccine used for primary vaccination
Ad26.COV2-S Updated:
J&J; Janssen Vaccine • 7/12/2021: Warning added to the EUA that adverse event reporting $10 per dose*
intramuscular injection 4/23/2021,
7/12/2021, suggests an increased risk of Guillain-Barré syndrome during the 42 days
10/20/2021 following vaccination
• 4/23/2021: EUA Fact Sheet for Recipients and Caregivers has been updated
to include information about the remote risk of blood clots
• Most people who developed these blood clots and low levels of
platelets were females ages 18 - 49 years of age; symptoms began
approximately one-twoweeks following vaccination
• Non-replicating adenovirus 26-vector dsDNA expressing SARS-CoV-2
spike protein
• No cost to patients
*Initially, the federal government will supply the vaccine at no cost.
CONTINUED
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November 2021
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 11/8/2021: Data from an ongoing Phase 3 trial of REGEN-COV reported a
reduction in the risk of contracting COVID-19 by 81.6% when given within
2–8 months after exposure
• 9/23/2021: The NIH updated its COVID-19 Treatment Guidelines to
recommend use of any one of the three authorized anti-SARS-CoV-2
mAbs to treat nonhospitalized patients with mild to moderate COVID-19
who are at high risk of clinical progression (REGEN-COV, sotrovimab, or
bamlanivimab/etesevimab)
• 7/30/2021: EUA was revised to authorize emergency use as post-exposure
prophylaxis for COVID-19 in adults and pediatric individuals (≥ 12 years of
age and weighing ≥ 40 kg) who are at high risk for progression to severe
COVID-19, including hospitalization or death
Original: $1,500 single dose
• REGEN-COV is not authorized for pre-exposure prophylaxis to prevent
REGEN-COV 11/21/2020 infusion
Treatment - COVID-19 before being exposed to the SARS-CoV-2 virus —only after
casirivimab + imdevimab Regeneron Updated: $450
monoclonal antibody exposure to the virus
intravenous infusion 6/3/2021, administration
7/30/2021 • 6/3/2021: Updates made to the EUA label include a change in the cost
authorized dosage (from 1200 mg of casirivimab and 1200 mg of
imdevimab to 600 mg of casirivimab and 600 mg of imdevimab), the
addition of a subcutaneous route of administration as an alternative to the
intravenous route, and the addition of a new REGEN-COV co-formulated
product in a single vial
• EUA is for casirivimab and imdevimab to be administered together for the
treatment of mild to moderate COVID-19 in adults and pediatric patients
(≥ 12 years of age and weighing ≥ 40 kg) with positive results of direct
SARS-CoV-2 viral testing, and who are at high risk for progressing to
severe COVID-19 and/or hospitalization
• No cost to patients, although healthcare facilities may charge fees
related to administration
*Initially, the federal government will supply the vaccine at no cost.
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November 2021
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 9/16/2021 - The FDA revised the EUA to include emergency use as post-
exposure prophylaxis for COVID-19 in adults and pediatric patients (≥ 12
years of age and weighing ≥ 40 kg) who are at high risk for progression
to severe COVID-19, including hospitalization or death; in this revision
of the EUA, bamlanivimab and etesevimab, administered together, are
authorized for use after exposure to the virus and are not authorized for
pre-exposure prophylaxis to prevent COVID-19 before being exposed to
the SARS-CoV-2 virus
• 9/23/2021: The NIH updated its COVID-19 Treatment Guidelines to
recommend use of any one of the three authorized anti-SARS-CoV-2
Original: mAbs to treat nonhospitalized patients with mild to moderate COVID-19
LY-CoV555 + LY- who are at high risk of clinical progression (REGEN-COV, sotrovimab, or
2/9/2021
CoV016 bamlanivimab/etesevimab)
Treatment -
bamlanivimab + Eli Lilly N/A
Revised: monoclonal antibody • 9/2/2021: Distribution of Eli Lilly’s bamlanivimab/etesevimab combination
etesevimab
6/25/2021, resumed in the United States following a pause due to prevalence of
intravenous infusion
9/16/2021 the Beta and Gamma variants; data show that the combination may be
ineffective against these 2 variants but retains activity against the Delta
variant, which is the dominant sustained variant in the United States
• EUA issued for the emergency use of bamlanivimab and etesevimab
administered together for the treatment of mild to moderate coronavirus
disease 2019 (COVID-19) in adults and pediatric patients ( ≥ 12 years of
age and weighing ≥ 40 kg) with positive results of direct SARS-CoV2 viral
testing, and who are at high risk for progressing to severe COVID-19 and/or
hospitalization
• Outpatient only - no cost to patients, although healthcare facilities
may charge fees related to administration
CONTINUED
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November 2021
EUA
Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC)
Date
• 9/23/2021: The NIH updated its COVID-19 Treatment Guidelines to
recommend use of any one of the three authorized anti-SARS-CoV-2
mAbs to treat nonhospitalized patients with mild to moderate COVID-19
who are at high risk of clinical progression (REGEN-COV, sotrovimab, or
bamlanivimab/etesevimab)
sotrovimab GlaxoSmithKline; EUA: Treatment - $2,100 single dose
• FDA granted an EUA for the treatment of mild-to-moderate COVID-19 in
intravenous infusion Vir 5/26/2021 monoclonal antibody infusion
adults and pediatric patients (≥ 12 years of age and weighing ≥ 40 kg) with
positive results of direct SARS-CoV-2 viral testing, and who are at high risk
for progression to severe COVID-19, including hospitalization or death
• Sotrovimab is not authorized for use in patients who are hospitalized due
to COVID-19
• 4/16/2021 - FDA revoked the EUA for bamlanivimab when administered
alone $1,250 single dose
Original:
LY-CoV555 infusion
11/9/2020 Treatment - • EUA was for the treatment of mild to moderate COVID-19 in adults and
bamlanivimab Eli Lilly $450
Revoked: monoclonal antibody pediatric patients with positive results of direct SARS-CoV-2 viral testing
intravenous infusion administration
4/16/2021 who are ≥ 12 years of age and weighing ≥ 40 kg, and who are at high risk for cost
progressing to severe COVID-19 and/or hospitalization
• 10/5/2021: JAMA study found that convalescent plasma treatment is futile
to treat critically ill COVID-19 patients
• The study found that the treatment resulted in a low likelihood of
Original:
U.S. Dept of providing improvement for severe COVID-19 patients who participated
Convalescent plasma 8/23/2020 Treatment - blood Unknown at this
Health and in the trial, but the research did not conclude why it was ineffective
intravenous infusion Revised: product time
Human Services • Current EUA is for the use of high titer convalescent plasma for the
2/4/2021
treatment of hospitalized patients early in the disease course and to those
hospitalized patients who have impaired humoral immunity and cannot
produce an adequate antibody response
CONTINUED
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November 2021
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 7/28/2021: EUA authorizes baricitinib as monotherapy for the treatment
of COVID-19 in hospitalized patients two years of age or older requiring
supplemental oxygen, non-invasive or invasive mechanical ventilation, or
extracorporeal membrane oxygenation (ECMO)
Original: • Under the revised EUA, baricitinib is no longer required to be $2,553 for 14-day
Olumiant administered with remdesivir (Veklury)
11/19/2020 course
baricitinib Eli Lilly Treatment - ARDS
Revised: • 11/19/2020: EUA for use in combination with Gilead’s Veklury (remdesivir) $91.17 per 2 mg
oral tablet
7/28/2021 for treating hospitalized patients with COVID-19 infection in patients tablet
aged two years of age or older, with suspected or laboratory confirmed
COVID-19 requiring supplemental oxygen, invasive mechanical ventilation,
or extracorporeal membrane oxygenation
• 4 mg once daily for 14 days or until hospital discharge
• EUA for the treatment of COVID-19 in hospitalized patients two years of
age and older who are receiving systemic corticosteroids and require
Actemra supplemental oxygen, non-invasive or invasive mechanical ventilation, or $3,228 for a single
Treatment - IL-6
tocilizumab Roche; Genentech 6/24/2021 extracorporeal membrane oxygenation (ECMO) infusion for a 70 kg
receptor antagonist
intravenous infusion patient
• 8-12 mg/kg single IV infusion; one additional infusion may be
administered
PIPELINE AGENTS
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 9/30/2021: Vaxzevria found to have 74% efficacy in preventing
symptomatic disease in the US, according to a report published in the New
Vaxzevria England Journal of Medicine
Currently Projected $3-$4
AZD 1222 AstraZeneca Vaccine
unknown • Non-replicating chimpanzee adenovirus-vector dsDNA expressing SARS- per dose*
intramuscular injection
CoV-2 spike protein
• No cost to patients
*Initially, the federal government will supply the vaccine at no cost.
CONTINUED
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November 2021
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 6/2021: NVX-CoV2373 was found to be 100% effective at protecting against
moderate and severe disease, according to findings from the Phase 3
NVX-CoV2373 PREVENT-19 trial Projected $10-$16
Novavax 2H 2021 Vaccine
intramuscular injection per dose*
• SARS-CoV-2 spike glycoprotein nanoparticle + adjuvant vaccine
• No cost to patients
• 11/5/2021: EUA request was submitted to the FDA for prevention of
COVID-19 in children 2-18 years of age based on results of an immuno-
bridging trial in pediatric patients which showed comparable neutralizing
Covaxin antibody response as in a large adult Phase 3 trial, the latter of which
reported 93.4% efficacy against severe COVID-19 disease, 77.8% overall No projected
BBV152 Ocugen 2H 2022 Vaccine
intramuscular injection efficacy, 63.6% efficacy against asymptomatic disease, and 65.2% efficacy pricing yet
against the Delta variant in nearly 25,800 adults
• Whole-virion, inactivated vaccine
• No cost to patients
• 9/9/2021: The FDA declined to approve the EUA for lenzilumab stating
it was unable to conclude that the known and potential benefits of
lenzilumab outweigh the known and potential risks of its use as a
treatment for COVID-19
• Humanigen expects an ongoing study to provide additional safety and
lenzilumab efficacy data to support its new EUA request No projected
Humanigen, Inc. 2022 Monoclonal antibody
intravenous infusion • 5/28/2021: EUA request submitted to the FDA, Lenzilumab achieved its pricing yet
primary endpoint with a 54% relative improvement in the likelihood of
survival without ventilation compared to placebo
• Lenzilumab is a proprietary first-in-class monoclonal antibody that
neutralizes GM-CSF, a cytokine of importance in the hyperinflammatory
cascade associated with COVID-19 and other indications
CONTINUED
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November 2021
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 10/14/2021: AstraZeneca submitted an EUA request to the FDA for
prophylaxis of symptomatic COVID-19. Top-line data from the Phase
III PROVENT trial showed that the treatment cut the risk of developing
symptomatic COVID-19 by a significant 77% compared to placebo
AZD7442
• The FDA submission also included results from the Phase III STORM
tixagevimab and No projected
AstraZeneca 2H 2021 Monoclonal antibody CHASER study involving participants recently exposed to the SARS-
cilgavimab pricing yet
CoV-2 virus, although that trial had failed to meet its primary endpoint of
intravenous infusion
preventing COVID-19 in these patients
• The monoclonal antibodies tixagevimab and cilgavimab bind to distinct
sites on the SARS-CoV-2 spike protein; they have been optimised with
half-life extension and reduced Fc receptor and complement C1q binding
• 10/14/2021: Merck filed for EUA. FDA advisory committee to meet
11/30/2021 to discuss safety & effectiveness of molnupiravir for the
treatment of mild-to-moderate COVID-19 in adults who have tested
positive and are at high risk for progression to severe illness.
• The FDA is not expected to issue a decision until December 2021 at the
earliest. Recent results from the Phase III MOVe-OUT trial showed that
Lagevrio molnupiravir halved the risk of hospitalisation or death versus placebo
Merck; Ridgeback No projected
molnupiravir 4Q 2021 Antiviral in at risk, non-hospitalised adults with mild-to-moderate COVID-19.
Therapeutics pricing yet
oral therapy Molnupiravir would become the first oral antiviral medication for
COVID-19, if cleared by the FDA
• Phase 2 - results showed that the percentage of outpatients who received
treatment were hospitalized and/or died less frequently vs. the placebo-
treated group
• Dose: oral twice daily for five days
CONTINUED
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November 2021
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 11/12/2021: In the Phase 3 PRESECO trial, favipiravir as a potential therapy
for patients with mild-to-moderate COVID-19 did not achieve statistical
favipiravir significance on the primary endpoint of time to sustained clinical recovery. Overseas priced at
Fujifilm; Others 2022 Antiviral Additional analyses of the trial data are ongoing
oral tablet $1 per tablet
• Dose: Loading dose twice daily on Day 1, then twice daily on
Days 2-7
• Interim results from the Phase 2/3 EPIC-HR trial demonstrated
significantly reduced hospitalization and death in non-hospitalized adult
patients with COVID-19 who are at high risk of progressing to severe
illness. The interim analysis showed an 89% reduction in risk of COVID-
19-related hospitalization or death from any cause compared to placebo
in patients treated within three days of symptom onset; 0.8% of patients
who received Paxlovid were hospitalized through Day 28 following
randomization (no deaths), compared to 7.0% of patients who received
placebo and were hospitalized or died (pCOVID-19
Pipeline Report
November 2021
Anticipated
Date of
Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC)
EUA or FDA
Approval
• 11/5/2021: FDA declined to issue an EUA, citing insufficient data to
establish a positive benefit-risk profile. NRx plans to supply the FDA with
data regarding at least 150 additional patients already treated in the
ACTIV-3b trial.
• 10/14/2021: NRx Pharma reported a 60-day survival rate of 81% of critical
COVID-19 patients suffering from respiratory failure who were treated with
Zyesami the drug Zyesami, compared to a survival rate of 21% among those who No projected
NRx Synthetic vasoactive
aviptadil 2H 2021 received standard care pricing yet;
Pharmaceuticals intestinal peptide
intravenous infusion available overseas
• 7/2021: NRx presented data from a randomized phase 2b/3 trial shows
patients treated with Zyesami are less likely to experience IL-6 cytokine
rise, and have improved survival and recovery from respiratory failure,
compared to patients receiving placebo. Data have been submitted to FDA
for EUA support
• 6/2021: EUA request resubmitted to the FDA after inital denial
© 2021 Envolve. All rights reserved. | Data is current as of 11/19/2021 Page 12Recent Specialty Drug Approvals
Pipeline Report
November 2021
Available at: AcariaHealth PANTHERx Rare Other
FDA
Drug Name Manufacturer(s) Indication(s) Approval Comments Cost (WAC)
Date
ENDOCRINOLOGY
• FDA-approved for the treatment of late-onset Pompe disease — it is the
second enzyme replacement therapy to be FDA-approved for Pompe
Nexviazyme disease, after Lumizyme
avalglucosidase
Genzyme; Sanofi Pompe disease 8/6/2021 • Prescribing Information includes black box warnings regarding severe $600,000/year
alfa-ngpt
hypersensitivity reactions, infusion-associated reactions, and risk of
intravenous infusion
acute cardiorespiratory failure in susceptible patients
• Projected impact: cost replacement of existing therapies
• Approved for the treatment of patients one year of age and older
Livmarli Cholestatic pruritus in
• There are currently ~2,000-2,500 children with ALGS in the U.S. who
maralixibat Mirum Pharmaceuticals patients with Alagille 9/29/2021 $391,000/year
would be eligible for treatment with maralixibat
oral solution syndrome (ALGS)
• Projected impact: cost increase
• Once-daily injection approved for use in children 5 years of age and
older whose growth plates are still open
Voxzogo
vosoritide • There are no FDA-approved agents for achondroplasia
BioMarin Achondroplasia 11/19/21 $328,000/year
subcutaneous • Injectable growth hormone products have been used off-label in a
injection limited number of cases
• Projected impact: cost increase
HEMATOLOGY
• Existing therapies for ANCA-associated vasculitis typically include
broad immunosuppression with daily doses of glucocorticoids such as
Anti-neutrophil prednisone or methylprednisone, followed by maintenance therapy with
Tavneos
cytoplasmic antibody a cyclophosphamide- or rituximab-based regimen
avacopan ChemoCentryx 10/7/2021 $176,000/year
(ANCA)-associated • Intended for use as an adjunct to existing therapies
oral capsule
vasculitis (AAV)
• Estimated U.S. prevalence: 55,000 people; ~15% have relapsed disease
• Projected impact: cost increase
1
Accredo, Mirum Access Plus, Amber, Hospital Administration, Pending Launch, Orsini
2 3 4 5 8
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November 2021
Available at: AcariaHealth PANTHERx Rare Other
FDA
Drug Name Manufacturer(s) Indication(s) Approval Comments Cost (WAC)
Date
IMMUNOLOGY
• Tissue-based regenerative therapy, intended for use once per lifetime
Rethymic • In the absence of treatment, children born with congenital athymia have
allogeneic processed no ability to fight infections and typically die within the first 24 months $2,729,500/one-
Enzyvant Congenital athymia 10/8/2021 of life
thymus tissue–agdc time treatment
surgical implant • Currently only available at Duke University Hospital
• Estimated prevalence: ~1 in 300,000 infants
NEPHROLOGY
• Approved for the treatment of moderate-to-severe uremic pruritus in
Korsuva Uremic pruritus CKD patients undergoing hemodialysis
difelikefalin Cara Therapeutics in chronic kidney 8/23/2021 Pending launch
• Administered at the end of each dialysis session
intravenous infusion disease (CKD)
• Projected impact: cost replacement of existing therapies
ONCOLOGY
• Approved for the treatment of VHL- associated renal cell carcinoma
(RCC), central nervous system hemangioblastomas, or pancreatic
neuroendocrine tumors, not requiring immediate surgery
• The Welireg Prescribing Information includes a black box warning
Welireg Von Hippel Lindau regarding embryo-fetal toxicity
belzutifan Merck (VHL)-associated 8/13/2021 $320,000/year
• Patients with VHL disease are at risk for benign blood vessel tumors as
oral tablet tumors
well as several cancers
• Estimated prevalence: 1 in 36,000 people (~10,000 cases in the U.S.); as
many as 70% of people with VHL disease develop RCC
• Projected impact: cost replacement of existing therapies
5
Pending launch, 6Biologics, 7Onco360
CONTINUED
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Pipeline Report
November 2021
Available at: AcariaHealth PANTHERx Rare Other
FDA
Drug Name Manufacturer(s) Indication(s) Approval Comments Cost (WAC)
Date
• Approved for the treatment of adult patients with locally advanced
or metastatic NSCLC with EGFR exon 20 insertion mutations, whose
Exkivity disease has progressed on or after platinum-based chemotherapy
Non-small cell lung
mobocertinib Takeda 9/15/2021 • Rybrevant is now FDA-approved for the same indication, with a higher $304,000/year
cancer (NSCLC)
oral capsule overall response rate and overall tolerability, albeit with an intravenous
route of administration
• Projected impact: cost replacement of existing therapies
• Approved for the treatment of adult patients with recurrent or
metastatic cervical cancer with disease progression on or after
Tivdak chemotherapy
tisotumab vedotin-tftv Genmab/Seagen Cervical cancer 9/20/2021 • Will compete with Keytruda in this indication $400,000/year
intravenous infusion
• FDA-approved with a black box warning regarding ocular toxicity
• Projected impact: cost replacement of existing therapies
• New indication for an existing CAR-T cell therapy
• Tecartus was previously FDA-approved only for the treatment of mantle
Tecartus cell lymphoma.
brexucabtagene Acute lymphoblastic • The new indication is for the treatment of adults with relapsed or $399,000/one-
Gilead 10/1/2021
autoleucel leukemia (ALL) refractory B-cell precursor ALL time treatment
intravenous infusion • Kymriah is similarly FDA-approved for ALL, but only for patients up to 25
years of age
• Projected impact: cost increase
• Approved for use in adult patients with Philadelphia chromosome-
positive CML (Ph+ CML) in chronic phase (CP), previously treated with
Scemblix two or more tyrosine kinase inhibitors, and for Ph+ CML in CP with the
Chronic myeloid T315I mutation
asciminib Novartis 10/29/2021 Pending launch
leukemia (CML)
oral tablets • Scemblix will compete with Iclusig (ponatinib), which is FDA-approved
for the same two indications
• Projected impact: cost replacement of existing therapies
4
Hospital Administration
CONTINUED
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Pipeline Report
November 2021
Available at: AcariaHealth PANTHERx Rare Other
FDA
Drug Name Manufacturer(s) Indication(s) Approval Comments Cost (WAC)
Date
• FDA-approved for the treatment of adults with PV
• Prescribing Information includes a black box warning re: fatal or life-
threatening neuropsychiatric, autoimmune, ischemic, and infectious
disorders
Besremi
• Designed for administration once every two weeks, followed by once
ropeginterferon
PharmaEssentia Polycythemia vera every four weeks during long-term maintenance
alfa-2b-njft 11/12/2021 Pending launch
Corporation (PV) • PV is a cancer originating from a disease-initiating stem cell in the
subcutaneous
injection bone marrow, estimated to affect more than 160,000 people in the
U.S., who have progressively burdensome symptoms; without proper
management, the disease progresses into malignancies including
myelofibrosis and acute myeloid leukemia
• Projected impact: cost replacement of existing therapies
6
Biologics, 7Onco 360, 9US Bioservices
© 2021 Envolve. All rights reserved. | Data is current as of 11/19/2021 Page 16Recent Non-Specialty Drug Approvals
Pipeline Report
November 2021
Drug Name Manufacturer(s) Indication(s) FDA Approval Date Comments Cost (WAC)
DERMATOLOGY
• Approved for the topical short-term and non-continuous
chronic treatment of mild to moderate AD in non-
immunocompromised patients ≥ 12 years of age whose
disease is not adequately controlled with topical
Opzelura prescription therapies or when those therapies are not $1,950/60 gm
ruxolitinib Incyte Atopic dermatitis (AD) 9/21/2021 advisable tube
topical cream
• Approved with a black box warning re: serious infections,
mortality, malignancy, MACE, and thrombosis (same
warnings that occur on the labels of oral JAK inhibitors,
even though Opzelura is a topical JAK inhibitor product)
NEUROLOGY
• FDA approved for the preventive treatment of episodic
migraine in adults
Qulipta
atogepant AbbVie Episodic migraines 9/28/2021 • Will compete with Nurtec ODT as the second oral calcitonin $12,000/year
oral tablet gene-related peptide (CGRP) receptor antagonist, as well
as with the injectable CGRP antagonists (Aimovig, Ajovy,
Emgality, Vyepti) in the migraine prophylaxis space
OPHTHALMOLOGY
• Administered as a preservative-free, aqueous nasal spray,
Tyrvaya while all other available agents for this indication are
Oyster Point Pharma, ophthalmic drops $296/4.2 mL
varenicline Dry eye disease 10/15/2021
Inc. bottle
nasal spray • Will comepte with Cequa, Restasis, and Xiidra in this
indication
• Once-daily eye drop
• Presbyopia is a progressive condition that reduces the eye's
Vuity ability to focus on near objects and usually impacts people
after age 40 $74/2.5 mL
pilocarpine AbbVie Presbyopia 10/28/2021
bottle
ophthalmic solution • U.S. prevalence is > 115 million people
• Currently eyeglasses and contact lenses are used for
treatment
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
CARDIOVASCULAR DISEASE
• Proposed for use in secondary prevention
patients with ASCVD
Atherosclerotic
• Dosed by subcutaneous injection administered
cardiovascular
Leqvio in the doctor’s office as a single dose every six
disease (ASCVD) PCSK9 synthesis
inclisiran Alnylam; Novartis months after an initial two doses given three $6,000/year 1/1/2022
and familial inhibitor
intravenous infusion months apart
hypercholesterolemia
(FH) • Would compete with Repatha and Praluent for
patients who are already on maximized statin
therapy
COAGULATION DISORDERS
• On August 19, 2020, BioMarin received a
Complete Response Letter for its BLA for
Roctavian* valoctocogene roxaparvovec $2 million/
valoctocogene roxaparvovec BioMarin Hemophilia A Gene therapy • The FDA recommended two years of data from one-time 4Q 2022
intravenous infusion the Company's ongoing Phase 3 trial in order to treatment
provide substantial evidence of a durable effect
• BLA resubmission is anticipated in 2Q 2022
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• For the treatment of adults with severe disease
(~40% of the total hemophilia B population)
• Current standard of care is factor IX replacement
therapy
• Recent data from the Phase 3 HOPE-B study
showed continued durable, sustained increases
in FIX activity at 52 weeks post-infusion with a
mean FIX activity of 41.5% of normal
• During the 52-week period, a single dose
AMT-061* significantly reduced the annualized rate of $1-2 million/
Uniqure; CSL bleeding requiring treatment by 80%
etranacogene dezaparvovec Hemophilia B Gene therapy one-time 2H 2022
Behring
intravenous infusion • The annualized rate of spontaneous bleeding treatment
requiring treatment was also significantly
reduced by 85% during the 52-week period
• Usage of FIX replacement therapy in all
patients declined 96%, with 52 of 54 patients
successfully discontinuing their prophylactic
infusions
• Estimated U.S. prevalence of hemophilia B:
~6,300 people; ~40% have severe disease
• Anticipated BLA filing: 1Q 2022
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Proposed for the treatment of adults with
severe disease (~60% of the total hemophilia A
population)
• Current standard of care is factor VIII
replacement therapy or Hemlibra
$1-2 million/
SPK-8011* • In the ongoing Ph I/II trial, factor VIII expression
Spark; Roche Hemophilia A Gene therapy one-time 2023
intravenous infusion was sustained in 16 of 18 participants with up to
treatment
four years of follow-up
• Across all dose cohorts, there was a 91.2%
reduction in annualized bleed rate
• There were no deaths and no FVIII inhibitor
development in the four years
• For the treatment of adults with severe disease
(~60% of the total hemophilia A population)
• This product is being studied in the Phase 3
AFFINE trial, which has been voluntarily paused
SB-525* Sangamo by the manufacturers to address the observation $1-2 million/
giroctocogene fitelparvovec BioSciences, Inc; Hemophilia A Gene therapy that some patients had factor VIII activity of one-time 2023
intravenous infusion Pfizer 150% or more, potentially raising their risk of treatment
blood clots. A study protocol amendment is
pending.
• Current standard of care is factor VIII
replacement therapy or Hemlibra
DERMATOLOGY
• Proposed for the treatment of the cutaneous
Filsuvez Keratinocyte manifestations of junctional and dystrophic EB,
Epidermolysis bullosa $100,000/
oleogel-S10 Amryt migration which is a rare genetic skin disorder that can 11/30/2021
(EB) year
topical ointment promoter cause skin to blister and tear from the slightest
friction or trauma
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Proposed for the treatment of moderate to
PF-04965842 severe disease in patients 12 years of age and
Janus kinase 1 older
abrocitinib Pfizer Atopic dermatitis $45,000/year 4Q 2021
(JAK1) inhibitor
oral tablet • FDA has indicated that its decision on abrocitinib
is delayed due to an ongoing safety review
ENDOCRINOLOGY
• Proposed for the treatment of hyperphagia and
LV-101 behavioral distress associated with PWS
Prader-Willi syndrome $250,000/
carbetocin Levo Therapeutics Oxytocin analog 12/1/2021
(PWS) • Would be the first FDA-approved therapy for year
intranasal spray
these PWS-related symptoms
• Racemic ketoconazole is already used off-label
for the treatment of Cushing's syndrome, but
Recorlev
Strongbridge Cortisol synthesis levoketoconazole is claimed to have improved $250,000/
levoketoconazole Cushing's syndrome 1/1/2022
Biopharma inhibitor efficacy, as it may inhibit hydroxylases more year
oral tablet
potently and reaches 3-fold higher plasma levels
in humans
• Proposed for the treatment of polyneuropathy of
hATTR amyloidosis in adults
ALN-TTRsc02 Transthyretin- TTR-targeting
vutrisiran Alnylam mediated (hATTR) RNA interference • Would compete with Onpattro (IV infusions every $400,000/ 4/14/2022
subcutaneous infusion amyloidosis agent three weeks) and Tegsedi (weekly subcutaneous year
injections) for the same indication, with quarterly
subcutaneous infusions
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
Cipaglucosidase
alfa: recombinant
human • Proposed for the treatment of late-onset Pompe
acid alpha- disease
AT-GAA* glucosidase
• Estimated U.S prevalence of Pompe disease: ~1
cipaglucosidase alfa/miglustat Amicus enzyme $500,000/
Pompe disease in 40,000 people 5/29/2022
intravenous infusion plus oral Therapeutics replacement year
therapy therapy (ERT) • Lumizyme and Nexviazyme are available FDA
approved ERT alternatives (Nexviazyme for late-
Miglustat: onset disease).
pharmacological
chaperone
HEMATOLOGY
• Proposed for the treatment of patients with
pacritinib CTI BioPharma
Myelofibrosis Kinase inhibitor intermediate and high-risk myelofibrosis with $175,000/year 11/30/2021
oral therapy Corp
low platelet counts of < 50,000/microliter
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Proposed for the treatment of adults with PKD
• The estimated prevalence of PK deficiency with
hemolytic anemia is ~3.3-12.2 per million in
Western populations; transfusion-dependent
patients account for 15-20% of those with PKD
• The Phase 3 ACTIVATE-T trial (n=27) in regularly
transfused adults demonstrated a statistically
AG-348 significant and clinically meaningful reduction
Agios Pyruvate kinase Pyruvate kinase in transfusion burden; in the 24-week fixed $250,000/
mitapivat 2/17/2022
Pharmaceuticals deficiency (PKD) stimulator dose period, 37% achieved a ≥ 33% reduction year
oral therapy
in transfusion burden compared to individual
historical transfusion burden, and 22% were
transfusion-free
• In the Phase 3 ACTIVATE trial in adults who do
not receive regular transfusions, 40% of patients
randomized to mitapivat achieved a hemoglobin
response compared to zero patients who
received placebo
Hypoxia-
inducible • Proposed for use in both dialysis- and non-
Vafseo dialysis-dependent CKD
factor prolyl
vadadustat Akebia/Vifor Anemia of CKD $13,000/year 3/9/2022
hydroxylase • Would compete with erythropoietin stimulating
oral tablet
inhibitor (HIF- agents (ESAs, e.g., Procrit, Aranesp)
PHI)
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Proposed for the treatment of patients with
beta-thalassemia who require regular blood
transfusions
• Demonstrated ability to dramatically decrease
or terminate the need for chronic blood
Beti-cel* Transfusion- transfusions $1-2 million/
betibeglogene autotemcel bluebird bio dependent beta- Gene therapy one-time 1H 2022
intravenous infusion thalassemia (TDT) • Twenty-five of 29 patients (86%) in a Phase 3 treatment
trial demonstrated transfusion independence
(TI) after treatment, with a median ongoing TI
duration of 26.3 months (min-max: 13.1 – 39.4)
• The BLA was submitted to the FDA in September
2021
INFECTIOUS DISEASE
SHP620 Post-transplant Viral DNA • Proposed for the treatment of both solid
$150,000/
maribavir Takeda cytomegalovirus synthesis organ transplant and hematopoietic stem cell 11/19/2021
year
oral tablet (CMV) infection inhibitor transplant recipients
• Proposed for the treatment of multi-drug
resistant HIV-1 infection in heavily treatment-
GS-6207 Human experienced (HTE) patients in combination with
HIV-1 capsid other antiretroviral agents $120,000/
lenacapavir Gilead immunodeficiency 2/28/2022
inhibitor year
subcutaneous injection virus (HIV)-1 • Would be the only HIV-1 treatment option
administered every six months
• Trogarzo has the same indication
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
MUSCULOSKELETAL CONDITIONS
• One-time treatment
• Three serious adverse effects were identified
in the Phase 3 CIFFREO trial, muscle weakness
including two cases of myocarditis, attributed to
the gene therapy
PF-06939926* $1-2 million/
Duchenne muscular • The study protocol is being amended to exclude
fordadistrogene movaparvovec Pfizer Gene therapy one-time 2023
dystrophy (DMD) patients with any mutation (exon deletion,
intravenous infusion treatment
exon duplication, insertion, or point mutation)
affecting exons 9-13, inclusive, or a deletion
that affects both exon 29 and exon 30; these
mutations are estimated to represent ~15% of
patients with DMD
• Would compete with SRP-9001 gene therapy for
GALGT2* those with mutations between exons 18 and 58 $1-2 million/
Sarepta
AAVrh74.MHCK.GALGT2 DMD Gene therapy • SRP-9001 is further along in the pipeline one-time 2023
Therapeutics
intra-arterial injection process, but comparative safety and efficacy are treatment
undetermined
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Targets exons 18-58 (~60-75% of DMD patients
have mutations in these exons)
• In October 2021 the global pivotal Phase 3
EMBARK trial was initiated
• Results from Study SRP-9001-101 (n=4, ages 4-7)
found that participants improved 8.6 points on
the North Star Ambulatory Assessment (NSAA)
compared to a matched natural history cohort
three years following a single administration of
SRP-9001 (pUpcoming Specialty Products
Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
NEPHROLOGY
• Proposed for the treatment of CKD caused by
Alport syndrome
RTA 402 • Estimated prevalence of Alport syndrome:
Reata CKD in Alport $150,000/
bardoxolone methyl Nrf2 activator ~10,000-60,000 people 2/25/2022
Pharmaceuticals syndrome year
oral capsule • Alport syndrome is estimated to account for 3%
of children with CKD and 0.2% of adults with
end-stage renal disease in the U.S.
NEUROLOGY
• Would compete with several other available
ARGX-113 Generalized therapies currently in use for gMG, such
$400,000/
efgartigimod argenx myasthenia gravis Anti-IgG antibody as cholineserase inhibitors, steroids, 12/17/2021
year
intravenous infusion (gMG) immunosuppressants, plasmapheresis, IVIG,
Rituxan and Soliris
• CDD is a serious and rare genetic disorder that
predominantly affects girls and is characterized
by early-onset, difficult-to-control seizures and
severe neuro‑developmental impairment
Seizures associated Positive allosteric • Most children affected by CDKL5 cannot walk,
ganaxolone Marinus talk, or care for themselves $150,000/
with CDKL5 deficiency modulator of 3/20/2022
oral therapy Pharmaceuticals year
disorder (CDD) GABA-A • Many also suffer from scoliosis, visual
impairment, gastrointestinal difficulties, and
sleeping disorders
• Currently, there are no approved therapies for
CDD
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• There are no approved therapies for the
treatment of AADC deficiency, which is an ultra-
rare enzyme deficiency disorder
• Estimated prevalence: ~5,000 patients
worldwide, with a live-birth incidence of
approximately 1 in 40,000 worldwide
• Five-year follow-up results from a clinical
trial show that motor function improvements
after PTC-AADC therapy were sustained,
demonstrating that the treatment effect is
durable
• Across three clinical trials, improvements
in motor development were recorded in all
PTC-AADC* Aromatic L-amino children from as early as three months $1-2 million/
eladocagene exuparvovec PTC Therapeutics acid decarboxylase Gene therapy one-time 2022
intraputamenal injection (AADC) deficiency • Cognitive and language skills were also treatment
reported to improve significantly from
baseline, as measured by Bayley-III scores,
with children able to understand their
caregivers and express themselves
• The rate of respiratory infection declined
from an average of 2.4 episodes per year at 12
months to 0.6 episodes per year at two years
and 0.3 episodes per year at five years
• Almost all treated children went from a
baseline weight below the third percentile to
making age-appropriate weight gains by 12
months following treatment
• Planned BLA submission in 1Q 2022
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Currently, the only therapeutic option for
patients with cALD is allogeneic hematopoietic
stem cell transplant (HSCT); beneficial effect has
been reported if performed early in the course of
cALD progression
• On 8/9/2021, bluebird bio announced that
one patient in the ALD-104 clinical trial had
developed myelodysplastic syndrome, and
that it was likely related to Lenti-D therapy; the
Lenti-D* Cerebral ALD-104 trial has been placed on clinical hold $1-2 million/
elivaldogene autotemcel bluebird bio drenoleukodystrophy Gene therapy pending further investigation into this case one-time 2H 2022
intravenous infusion (cALD) treatment
• In the U.S., newborn screening for ALD has been
added to the Recommended Universal Screening
Panel and is currently active in 20 states,
accounting for ~60% of U.S. newborns
• The worldwide incidence of ALD is ~1 in 5,000 to
1 in 17,000 newborns (both male and female),
and ~1 in 20,000 to 1 in 30,000 newborn males;
cALD develops in approximately 40% of affected
boys and in a smaller number of adult men
ONCOLOGY
• Proposed for the treatment of relapsed/
refractory disease after at least three prior lines
JNJ-68284528 of therapy $450,000/
Anti-BCMA
ciltacabtagene autoleucel Janssen Multiple myeloma • Demonstrated a 97% overall response rate and one-time 2/28/2022
CAR-T therapy
intravenous infusion 67% achieved a stringent complete response at treatment
a median follow-up of 12.4 months in the Phase I/
II CARTITUDE-1 trial
*Expected to cost > $500,000 per member.
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• New indication for an existing CAR-T cell therapy
• Proposed for the treatment of adults with
relapsed or refractory FL after two prior lines of
treatment
• In the pivotal Phase II ELARA trial, 66% achieved
Kymriah a complete response and the overall response $475,000/
Follicular lymphoma
tisagenlecleucel Novartis CAR-T therapy rate was 86% in heavily pretreated patients; no one-time 2/27/2022
(FL)
intravenous infusion patients experienced grade 3 or higher cytokine treatment
release syndrome related to Kymriah within the
first eight weeks following infusion
• Yescarta is another CAR T-cell therapy that is
FDA-approved as 3rd-line treatment for relapsed
or refractory FL
Immunoglobulin
Tyvyt • Proposed for use in combination with
Non-small cell lung G4 (IgG4)
sintilimab Eli Lilly pemetrexed and platinum chemotherapy for $175,000/year 3/18/2022
cancer (NSCLC) monoclonal
intravenous infusion first-line treatment of nonsquamous NSCLC
antibody
• Proposed for use in combination with umbralisib
Chronic lymphocytic (Ukoniq)
TG-1101 Anti-CD20
leukemia (CLL) and $125,000/
ublituximab TG Therapeutics monoclonal • It is estimated that there are approximately 3/25/2022
small lymphocytic year
intravenous infusion antibody 20,000 new cases of CLL diagnosed each year in
leukemia (SLL)
the United States
HMPL-012 • Proposed for the treatment of advanced and
Hutchison China Neuroendocrine Tyrosine kinase $200,000/
surufatinib progressive pancreatic and extra-pancreatic NETs 4/30/2022
MediTech Limited tumors (NETs) inhibitor year
oral therapy in patients who are not amenable for surgery
• Proposed for use in combination with nivolumab
BMS-986016
Bristol Myers LAG-3-blocking (Opdivo) for the treatment of adult and pediatric
relatlimab Melanoma $175,000/year 3/19/2022
Squibb antibody patients (≥ 12 years of age and weighing ≥ 40 kg)
intravenous infusion
with unresectable or metastatic melanoma
CONTINUED
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Pipeline Report
November 2021
Anticipated
Mechanism(s) Anticipated
Drug Name Manufacturer(s) Indication(s) Comments Approval
of Action Cost
Date
• Proposed for use in the post androgen receptor
pathway inhibition, post taxane-based
chemotherapy setting
Metastatic castration- Targeted • In the pivotal, Phase III VISION study 177Lu-
Lu-PSMA-617
177
$150,000/
Novartis resistant prostate radioligand PSMA-617 plus standard of care significantly 1H 2022
intravenous infusion year
cancer (mCRPC) therapy improved overall survival and radiographic
progression-free survival for men with progressive
PSMA-positive mCRPC compared to standard of
care alone
Humanized • Proposed for the treatment of patients with
BGB-A317
IgG4 anti-PD-1 unresectable recurrent locally advanced or
tislelizumab BeiGene/Novartis Esophageal carcinoma $175,000/year 7/12/2022
monoclonal metastatic esophageal squamous cell carcinoma
intravenous infusion
antibody after prior systemic therapy
RESPIRATORY
• Proposed for the treatment of severe uncontrolled
asthma in adults and adolescents
Anti-thymic • Would compete with Dupixent, Cinqair, Nucala,
stromal Fasenra;
AMG157
AstraZeneca; lymphopoietin • All of these, however, except Dupixent, are
tezepelumab Asthma $42,000/year 1/7/2022
Amgen (TSLP) only approved for the eosinophilic subtype
subcutaneous injection
monoclonal • Dupixent is also approved for those who are
antibody oral corticosteroid-dependent
• Non-eosinophilic disease accounts for around half
of severe asthma cases
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